Sirnaomics Received IND Approval from CFDA for Its Leading siRNA Therapeutic Candidate, STP705, for Treatment of Hypertrophic Scar

Tuesday, May 02, 2017

Sirnaomics, Inc., a leading biopharmaceutical company in discovery and development of RNAi therapeutics, announces today that its subsidiary company, Suzhou Sirnaomics Pharmaceuticals, Co. Ltd, China, has received an approval from China Food and Drug Administration (CFDA) for an IND application of STP705 (Cotsiranib®), an siRNA (small interfering RNA) therapeutic for treatment of hypertrophic scar. The IND was filed through a chemo-drug category 1.1 application based on CFDA requirement. The approval represents a milestone event for the company and, more importantly, it marks the first in the country allowing innovative siRNA therapeutics to enter clinical study, with the IND filed from a domestic company.

Suzhou Sirnaomics' lead product candidate, STP705, is an anti-fibrosis siRNA therapeutics taking advantage of a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery, to directly diminish both fibrotic activity and inflammatory activity allowing for application in many disease states. This initial clinical study is designed to evaluate safety and tolerance of STP705 through healthy volunteers with both single dose escalation and multiple dose escalations.

"The green light from CFDA for our STP705 IND submission represents another major milestone following the US FDA approval of such a clinical study five months ago. It has demonstrated that Sirnaomics' team is able to build platforms achieving preclinical study success and overcoming the regulatory hurdles in both countries, for discovery and development of novel siRNA therapeutics," said the Chairman of the board of Suzhou Sirnaomics, Dr. Patrick Y. Lu. "With experience and resources from both countries, our team is even more confident in development of not only STP705 but an enriched pipeline of siRNA therapeutic candidates, to address various clinical unmet needs on the two largest pharmaceutical markets in the world."

"The treatment of abnormal scarring is very important to our patients and the condition can result in both physical and psychological distress. There is a clear unmet medical need in the current treatment of patients with hypertrophic scars and it is very exciting to see new therapeutics such as STP705 enter into clinical study to address a problem that currently has very few effective treatment options," stated Mr. Qing-Feng Li, MD, Ph.D., Professor of Shanghai Jiao-Tong University and the 9th Hospital of Shanghai, a nationally renowned expert for plastic surgery.

"We are very excited to see that STP705 IND has received an approval from CFDA. This is a demonstration of a successful partnership between Xiangxue Pharmaceuticals and Sirnaomics, and the complimentary strength of both companies in development of novel siRNA therapeutics. Currently, we are more than ready for moving ahead STP705 into clinic and serving for those patients with unmet clinical needs," said Dr. Lun Zeng, Senior Vice President of Xiangxue Pharmaceuticals, Co. Ltd., Guangzhou, China.

About the Planned Clinical Study

This approved clinical study is staged into 2 parts: a randomized, double-blind placebo controlled study to evaluate the safety of STP705 administered as subcutaneous injection into healthy volunteers with either single dose escalation or multiple dose escalation. Both pharmacokinetics and pharmacodynamics of STP705 will be evaluated throughout this study.

About Hypertrophic Scar

Hypertrophic scar formation is a major clinical problem in the developing and industrialized worlds. Burn injuries, traumatic injuries, and surgical procedures can give rise to exuberant scarring that result in permanent functional loss and the stigma of disfigurement. Hypertrophic scar's form as a result of aberrations of physiologic wound healing and may arise following any insult to the deep dermis. By causing pain, pruritus and contractures, excessive scarring significantly affects the patient's quality of life, both physically and psychologically. Pathophysiology of hypertrophic scars entails a prolonged inflammatory and proliferative phase of wound healing after injury. Among various cytokines promoting hypertrophic scar formation TGF-β1 is known as a key regulator of the aberrant fibrogenic response, while COX-2 as a potent proinflammatory and proliferative mediator.

About STP705

STP705 is composed of two siRNA oligonucleotides, targeting TGF-β1and COX-2 mRNA respectively, and formulated in nanoparticles with Histidine-Lysine Co-Polymer (HKP) peptide. Each individual siRNA was demonstrated to inhibit the expression of their target mRNAs and combining the two siRNA's produced a synergistic effect that diminished pro-fibrogenic factors. Molecular analyses of the effects of administering the combination demonstrated that the inhibition of these targets had effects on downstream gene products associated with fibrosis including: α-SMA, Col1A1, and Col3A1. Additional data suggests that reductions in TGF-β1 and COX-2 led to proapoptotic effects in fibroblasts. This data also suggests that STP705 has the potential for broad application in many inflammatory and fibrotic driven disease states. A Phase IIa clinical study of STP705 for hypertrophic scar treatment is ongoing in United States.

About Suzhou Sirnaomics Pharmaceuticals Co., Ltd.

Suzhou Sirnaomics is a subsidiary of Sirnaomics, Inc., a leading biopharmaceutical company for discovery and development of RNAi therapeutics, headquartered in Gaithersburg, Maryland, USA. The company's mission is to alleviate human suffering and advance patient care in areas of high unmet medical need. Members of the senior management team have a great deal of combined experience in the biopharmaceutical, clinical, financial and business management in both USA and China. The company is supported with funding from private investors, corporate partnerships and government grants. As the R&D center in China, Suzhou Sirnaomics has developed a strong portfolio of intellectual properties with an enriched product pipeline. The therapeutic areas of interest include anti-fibrotic and anti-inflammatory disease states, cancer therapeutics, and others.